Patent SG-11201804070X-A (SG)
Title: Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
Inventor: Falkner Falko-Günter
IPC: C12N15/86
Filed: 2016-11-11
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property - Organization International Bureau rezt.dlid (43) International Publication Date .... .....r ....) 18 May 2017 (18.05.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111111111111111111111111 2017/083762 Al (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every C07K 14/755 (2006.01) C12N 15/86 (2006.01) kind of national protection available): AE, AG, AL, AM, A61K 48/00 (2006.01) AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, (21) International Application Number: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/US2016/061684 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, (22) International Filing Date: KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, 11 November 2016 (11.11.2016) MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, (25) Filing Language: English SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, (26) Publication Language: English TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 62/255,317 13 November 2015 (13.11.2015) US (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (71) Applicants: BAXALTA INCORPORATED [US/US]; GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 1200 Lakeside Drive, Bannockburn, IL 60015 (US). BAX- TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, ALTA GMBH [CH/CH]; Thurgauerstrasse 130, CH-8152 TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, Glattpark (Opfikon) (CH). DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, (72) Inventor; and LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, (71) Applicant : LENGLER, Johannes; Schrotzbergstrasse SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, 2/23, 1020 Vienna (AT). GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: (72) Inventors: FALKNER, Falko-Giinter; Neusiedlzeile be 76A, 2304 Orth/donau (AT). HORLING, Franziska; — as to applicant's entitlement to apply for and granted a Bauemfeldgasse 3/9, 2230 Gaenserndorf (AT). ROTTEN- patent (Rule 4.170) STEINER, Hanspeter; Haidgasse 10/17, 1020 Vienna as to the applicant's entitlement to claim the priority of the SCHEIFLINGER, Friedrich; Michelbeuemgasse (AT). earlier application (Rule 4.17(iii)) 4/17, 1090 Vienna (AT). Published: Agents: ANTCZAK, Andrew, MORGAN, (74) J. et al.; — with international search report (Art 21(3)) LEWIS & BOCKIUS LLP, One Market, Spear Street Tower, San Francisco, CA 94105 (US). — listing description 5.2(a)) with sequence part of (Rule (54) Title: VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A Il Human wild-type RAH .4t :ppaz ,, ;;; ;;Fr;; EIPA;;;M ei Ir-- Refacto-type BDD-FVIII ,---- M ;' ,B;; / / / M j/ rALLfAN Ettf5 / 211 -r 2 GO 0 Figure 1 Il O (57) : The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and meth - 0 ods for treating hemophilia A.